Articles tagged with Gene Therapy
Researchers have built a new model to examine Usher Syndrome, a leading cause of combined deafness and blindness. The model replicates the visual problems not addressed by previous models, offering insight into strategies for designing therapeutic interventions.
Researchers at Tokyo Medical and Dental University have found that a specific lipid, alpha-tocopherol, increases the uptake of antisense oligonucleotides in the stroke-lesioned brain. This delivery method has potential for targeted protein expression after a stroke.
Researchers at the University of Tokyo have discovered the 3D structure of TnpB, a protein involved in genome editing and a probable precursor to the CRISPR-Cas12 enzyme. The study reveals how TnpB recognizes and cuts DNA using a unique pseudoknot shape similar to that found in guide RNAs of Cas12 enzymes.
Researchers at Indiana University School of Medicine investigated the protein BVES and its crucial role in muscular dystrophy. They discovered that inhibiting proteasome degradation with Bortezomib can alleviate symptoms of BVES-deficient muscular dystrophy.
Researchers at Children's Hospital of Philadelphia discovered that viral proteins use phase separation to coordinate the complex process of replicating viral genomes and then encapsulating them in a viral particle. This process allows for the orderly and coordinated formation of infectious viral offspring.
A recent study found that a tiny fragment of the PC1 protein can suppress the disease in mouse models, reducing cyst size and revealing new avenues for developing therapies. The researchers discovered that this small piece interacts with a mitochondrial protein called NNT, opening up new avenues to study its normal function.
Researchers developed a programmable bacterial injection system that can deliver a range of proteins, including those for gene editing, to different cell types. The system has shown promising results in targeting cancer cells and delivering proteins to the brain in live mice without provoking an immune response.
The NCATS Platform Vector Gene Therapy (PaVe-GT) program utilizes FDA guidance to prepare applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), stimulating gene therapy development. The PaVe-GT program has already helped receive ODD and RPDD designations for a gene therapy product treating PCCA-r...
Researchers have found that valosin-containing protein (VCP) is essential for KRAS-mutant pancreatic ductal adenocarcinoma cell growth and survival. Inhibiting VCP, combined with autophagy inhibition, enhances efficacy in preclinical studies.
Researchers at Tokyo Medical and Dental University have developed a polymeric nanoparticle gene delivery system that promotes bone formation after traumatic inflammation. The therapy inhibits excessive inflammation and prevents residual ridge resorption, leading to improved tissue healing after tooth extraction.
Researchers at Texas A&M University have developed the first molecular therapeutic for Angelman syndrome, a devastating neurogenetic disorder. The therapy targets an evolutionarily conserved region in the UBE3A-AS transcript and has shown promising results in clinical trials.
Researchers used a monoclonal antibody to reduce circulating IgG levels, allowing for successful gene delivery to the liver and heart via systemic AAV-based gene therapy in mice and non-human primates. The strategy has potential to circumvent neutralizing antibody limitations and expand gene therapy accessibility.
Researchers at Harvard University developed a novel RNA sense-and-respond circuit, DART VADAR, which utilizes an enzyme to detect specific molecular markers of disease and cell types. This enables highly specific treatments for various diseases by triggering the translation of therapeutic genetic payloads.
Researchers at UCLA successfully used base editing to correct a mutation causing rare immune deficiency CD3 delta SCID. The treatment corrected an average of 71% of patient stem cells and allowed them to produce fully functional T cells, suggesting long-term persistence of corrected blood stem cells.
A new study published in Blood Advances found that the average life expectancy of publicly insured individuals with sickle cell disease is roughly 52.6 years, which is significantly lower than the overall US life expectancy of 73.5 years for men and 79.3 years for women. The study also revealed worse survival outcomes among those insur...
Researchers have designed a new RNA sensor that can selectively activate synthetic genes in specific cells, opening up possibilities for targeted therapies for cancer and other diseases. The system uses an enzyme that naturally exists in most animal cells to detect and repair mismatches in double-stranded RNA.
Researchers have successfully used AAV1.NT-3 gene therapy to improve muscle physiology and prevent age-related sarcopenia in mice. The treatment resulted in restored muscle mass, strength, and neural connections, offering a potential new option for managing this debilitating condition.
Pancreatic cancer is an incurable form of cancer, but gene therapies are being developed to treat it. The article reviews ongoing clinical trials and discusses various biotherapies, including vaccines and CAR-T cell therapy.
Gene therapy using CRISPR-Cas9 lipid nanoparticles has been shown to be highly effective in reducing target protein expression in mice. The new delivery system increases the efficiency of in vivo gene therapy, paving the way for safe and effective treatment.
Researchers found that severe herpesvirus infections can strongly activate host cellular immunity, leading to a therapeutic effect on refractory adult T-cell leukemia/lymphoma. This activation may play an important role in the survival of patients with this intractable disease.
Researchers have developed a new gene therapy to treat alpha 1-antitrypsin deficiency, an autosomal recessive disorder that causes emphysema. The therapy uses an adeno-associated virus (AAV) vector coding for an oxidation-resistant human α1-antitrypsin protein.
Researchers at UTHSC are working on a project to find the first therapeutic intervention to prevent frontotemporal dementia or slow its progression in a mouse model linked with the condition. They aim to use DNAzymes to target pathological tau aggregates, which cause cognitive impairment and progressive neuropathological symptoms.
A recent study published in Immunity reveals that human T-cell receptor genes exhibit unexpectedly high variability among individuals, with each person having a unique set of gene variants. The researchers identified 175 new gene variants originating from Neanderthals, which are present in up to 20% of modern humans in Europe and Asia.
Researchers from Rice University have developed a new approach to control gene expression using proteolysis targeting chimeras (PROTACs). By reengineering the PROTAC molecular infrastructure, they demonstrated the ability to achieve chemically induced dimerization (CID), allowing for precise control over gene activation in specific loc...
Researchers evaluate an integrated NGS system, delivering accurate diagnoses in under 24 hours and expanding targeted treatments available to patients with myeloid neoplasms. The assay identified 80-92% of genetic variants, demonstrating promising results for accelerating precision therapies.
Scientists have identified genes that play key roles in the development of coronary artery disease (CAD), a leading cause of death worldwide. The study found notable differences in gene activity between males and females, as well as between cells that were multiplying and those that were not.
Researchers from HKUMed discovered that somatic deletion of AKTIP promotes luminal breast cancer development and resistance to endocrine therapy. Blocking the alternative escape pathway with a JAK2/STAT3 inhibitor can overcome this resistance, offering new therapeutic possibilities for patients with AKTIP gene deletion.
Researchers discover gene therapy ophNdi1 that boosts mitochondrial performance in retinal ganglion cells, potentially treating glaucoma and age-related macular degeneration. The therapy shows protective effects in three models of mitochondrial dysfunction.
A novel form of chemoimmunotherapy has been proven to be a promising treatment for canine cancer, extending the lives of terminally ill dogs. The therapy uses modified Mesenchymal Stem Cells carrying a potent 'kill-switch' that induces anti-cancer immunity and improves quality of life.
Researchers have discovered an anti-ageing gene that can rewind the heart's biological age by 10 years, offering a potential target for patients with heart failure. The study found that administering the healthy mutant gene to elderly patients with severe heart problems improved their cardiac function and rejuvenated their immune system.
Researchers at Children's Hospital of Philadelphia have developed a custom-built application to automate the determination of engraftment after hematopoietic stem cell transplant. The tool has been shown to improve accuracy of reported engraftments, reducing errors in neutrophil and platelet engraftment reporting.
Researchers at Massachusetts General Hospital created a new class of technologies called CRISPR-associated transposases (CASTs) to overcome diverse disease-causing mutations. The optimized approach improves product purity and genome-wide specificity, offering a potential solution for inserting entire genes into the genome.
Researchers found four genetic populations of the finless porpoise in Chinese waters and three in the Gulf of Thailand, with unique matriline lineages. The study suggests ongoing gene flow among populations, requiring careful evaluation of inter-population connectivity for conservation action.
Researchers at University of Texas M. D. Anderson Cancer Center develop a novel mRNA delivery system using extracellular vesicles, which can initiate collagen production in cells and has potential for other mRNA therapies
A new approach to gene therapy for inherited blindness uses lipid nanoparticles to deliver mRNA inside the eye, targeting light-sensitive cells and creating proteins that edit vision-harming gene mutations. The technology has shown promising results in animal studies, including mice and nonhuman primates.
A new gene therapy treatment has shown promising results in a clinical trial for Artemis-SCID patients, improving their immune function and reducing treatment complications. The treatment involves adding a healthy copy of the Artemis gene to the patient's own cells, resulting in improved T-cell and B-cell immunity.
The use of 3D-patient tumor avatars (3D-PTAs) is crucial for guiding treatment decisions in precision oncology. These avatars, including patient-derived organoids, 3D bioprinting, and microscale models, can accurately depict a tumor with its microenvironment, enabling the testing and prediction of therapeutic drug efficacy.
Scientists at Duke University have made a breakthrough in controlling gene expression in response to injury, using a segment of fish DNA called TREE. The method successfully targeted gene activity to specific regions and time windows, showing promise for regenerating damaged tissues in mammals.
Researchers from Japan have developed an RNA interference method using antisense oligonucleotides to correct a genetic defect in Fukuyama Muscular Dystrophy. This approach has shown promise in treating patients with the disease, which is characterized by generalized muscle weakness and intellectual disability.
A study by Sanford Burnham Prebys found that misfolded proteins in liver cells contribute to liver cancer development, potentially increasing the risk of certain patients receiving gene therapies for hemophilia. Improving FVIII folding could decrease this risk and develop safer treatments.
Researchers have discovered that a specific mutation in the misfolding protein causing Parkinson's disease can also protect against multiple system atrophy (MSA), another fatal neurodegenerative disorder. The findings provide a promising lead for developing targeted treatments using personalized medicine approaches.
Researchers have developed an AAV-based gene therapy to treat glutaric aciduria type I (GA-I), a rare genetic disorder causing accumulation of glutaric acid in the central nervous system. The treatment has shown promising results, with a survival rate of 100% in mice treated intracerebroventricularly.
Researchers at University of Pittsburgh have designed novel nanoparticles that co-deliver a chemotherapy drug and a novel immunotherapy, shrinking tumors in mouse models of colon and pancreatic cancer. The therapy silences a gene involved in immunosuppression by blocking Xkr8 protein distribution on the cell membrane.
Nucleic acid therapies aim to treat genetic disorders and diseases, but delivering therapeutics is a significant challenge. Researchers are investigating nanoparticle delivery systems to target specific cells and sub-cellular compartments for effective delivery.
Researchers have engineered a family of adeno-associated viral vectors that can deliver cargo to the primate brain, offering a safer and more efficient way to treat genetic diseases. The PAL family of AAVs has been shown to be three times better at delivering their cargo into the brain than current leading AAV delivery vehicle AAV9.
The UC San Diego Alpha Stem Cell Clinic will receive $8 million in funding to launch new clinical trials and improve accessibility of stem cell therapies. The clinic has already launched 59 clinical trials and treated 277 patients with various diseases.
The USC+CHLA Alpha Clinic will advance clinical trials of new cell and gene therapies, engaging underserved communities and training the workforce that conducts them. This program complements existing efforts to translate discoveries into new therapies for better health outcomes.
Researchers found that IGF1 gene therapy increases kisspeptin expression and GnRH release, and alters microglial cell numbers, suggesting a potential protective effect against reproductive decline. This could lead to new strategies for optimizing lifespan and combating age-related health problems in women.
A Rice University bioengineer has developed a noninvasive technology to measure gene expression in deep tissues, particularly in the brain. This innovation could improve the monitoring of gene therapy treating neurodegenerative disorders such as epilepsy, ALS, and Huntington's disease.
Researchers have developed a new gene therapy that selectively targets overactive brain cells, reducing excitability and suppressing seizures in mice. The treatment shows promise for treating neurological disorders such as epilepsy, Parkinson's disease, schizophrenia, and pain disorders.
Amanda Woerman's research aims to disrupt tau misfolding, a common thread in fatal neurodegenerative disorders PSP and CBD. The grant-funded study seeks to test a proof-of-concept gene therapy for these conditions, potentially leading to personalized medicine breakthroughs.
The Alpha Stem Cell Clinic will develop preclinical studies into early and later phase clinical trials with the goal of establishing advanced regenerative medicine treatments. The clinic will also foster greater collaboration with eight similar clinics across the state and educate the public about stem cell and related therapies.
Johns Hopkins Medicine researchers have developed a novel genetic engineering approach to deliver gene therapy by utilizing a cell's natural process to
A new Northwestern Medicine study identifies common and rare gene mutations that impact radiation resistance and sensitivity. This information will allow clinicians to better calibrate radiation doses based on genetic mutations, improving treatment efficacy while reducing toxicity.
Researchers at the University of Pennsylvania School of Medicine have developed a gene therapy that rapidly restores night vision in adults with LCA, a common form of congenital blindness. The treatment has been shown to improve rod-based visual functions and correct severe visual deficits in patients.
Researchers at CNIO have identified alveolar type II pneumocytes as the primary cell type responsible for developing pulmonary fibrosis. The study reveals that targeting these cells through telomere-based therapy may lead to a breakthrough in treating this debilitating disease.
Nanoscope Technologies received a $1.5M NIH SBIR grant to develop gene-agnostic glaucoma therapy utilizing Engineered Mechanosensitive Channels (EMCs). EMCs can autonomously regulate pressure, offering a novel approach to treating Glaucoma.
Researchers have used single-cell sequencing to uncover novel gene expression patterns in injured kidney cells, providing new avenues for biomarker discovery and treatment. The studies reveal that epithelial cells of all tubule segments are involved in the injury processes, with distinct molecular patterns across patients.
Researchers at Universidad de Navarra identified a biomarker that predicts CAR T cell therapeutic capacity, which could improve treatment outcomes for patients. The study found that high CAR density in CAR T cells is associated with a worse clinical response in hematological tumors.
Researchers developed a computational platform to identify metabolic vulnerabilities in ovarian cancer genes, suggesting opportunities for targeted therapies. The study found that certain genetic alterations can create vulnerabilities in cancer cell metabolism, which can be exploited to selectively kill cancer cells.